A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting (HomERT)

Study Overview

Primary objective:

To obtain data pertaining to the safety and tolerability of alglucosidase alfa and laronidase treatments administered in a home-care infusion setting.

Secondary objectives:

  • To evaluate personal satisfaction of both cohorts of patients treated in a home-care infusion setting.
  • To evaluate the infusion compliance in both cohorts of patients treated in a home-care infusion setting.

Study details

Prospective observation duration for each patient: at least 12 months (from enrollment)

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Pompe Disease, Mucopolysaccharidosis Type I (MPS I)
  • Gender: All

Inclusion Criteria:

  • Signed, informed consent obtained prior to being enrolled into the study and prior to starting any data collection. Consent of a legally authorized guardian is required for legally minor patients as defined by local regulation. If the patient is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from the patient, if applicable.
  • Pompe disease patients with confirmed acid alpha-glucosidase (GAA) enzyme deficiency treated with Myozyme® in home infusion setting according to authorized clinical practice and the approved risk management plan document (Cohort A) or
  • MPS I patients with confirmed deficiency of the lysosomal enzyme, alpha-L-iduronidase treated with Aldurazyme® in home infusion setting according to authorized clinical practice and the approved risk management plan document (Cohort B).

Exclusion Criteria:

  • Participation in another clinical trial with any investigational agent within the 12 weeks preceding enrolment.
  • Any condition (e.g. medical concern) which, in the opinion of the Investigator, would make the participant unsuitable for the study.
        The above information is not intended to contain all considerations relevant to a potential
        participation in a clinical trial.

Updated on 14 Feb 2024. Study ID: NCT05073783